On May 14, 2021 Kiromic BioPharma (NASDAQ: KRBP), a pre-clinical stage biotechnology company using its proprietary DIAMOND artificial intelligence ("A.I.") platform to improve drug discovery and development with a therapeutic focus on immune-oncology, reported its quarterly results for the three months ended March 31, 2021, and provided an update on its corporate developments.

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"We are thankful to our employees and collaborators who have maintained this high level of execution this year. From their efforts, we plan to submit two investigational new drug applications to the United States Food and Drug Administration by the end of May 2021."

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"Kiromic BioPharma achieved important scientific and operational milestones during the year that we believe have us well positioned for preparing our staff and our facilities for the first in-human dosing in Q3 2021," said Dr. Maurizio Chiriva-Internati, PhD, CEO and President of Kiromic BioPharma. "We are thankful to our employees and collaborators who have maintained this high level of execution this year. From their efforts, we plan to submit two investigational new drug applications to the United States Food and Drug Administration by the end of May 2021."

Our approach and goal are to defeat cancer by developing immunotherapies by improving target discovery and validation. With better targets, we believe our therapies will be more effective than the current array of immunotherapies using older targets.

Corporate and Scientific Highlights

Events Occurring during the three months ended March 31, 2021

Facility Expansion in Houston, TX – On March 22, 2021, we executed a lease expansion within our premises in Houston, TX. The amended lease agreement will commence on August 1, 2021 under an operating lease agreement that is noncancelable from commencement until May 1, 2024. The amended lease agreement adds approximately 15,385 square feet to the current facility. Total square feet will be approximately 38,223 square feet.
Leon Office, Asia, Strategic Marketing Agreement – On January 28, 2021, we executed a strategic alliance agreement with Leon Office, Asia (H.K.) ("Leon") a company established under existing laws of Hong Kong. Leon will act as an independent business development advisor on the behalf of the Company. Leon will seek to introduce organizations and individuals that will create business development opportunities for the Company, to expand the Company’s reach to international markets with a focus on certain Asian markets and to increase brand recognition and exposure through developing liaisons, collaborations, branches and subsidiaries. They will also use commercially reasonable efforts to research the Asian market, with a primary, but not exclusive, focus on determining the most suitable structures for the development of medical partnerships or joint ventures with scientific partners in the Asian market with a mission to test products to be created by the joint venture resulting from such partnership and to develop validation programs for any products produced by such joint venture, including programs for clinical trials and human testing and, ultimately, for product certification. The cost of the agreement is $360,000 annually, payable in four quarterly installments.
SBA Loan Extinguishment – On February 16, 2021 the Small Business Administration ("SBA") granted forgiveness of our SBA loan and all applicable interest. On the date of forgiveness, the principal and accrued interest totaled $105,800. The forgiveness was classified as a gain on loan extinguishment in the consolidated statement of operations.
Events occurring after March 31, 2021 until May 14, 2021

Research Grant Agreement with University of Texas MD Anderson Cancer Center – On April 8, 2021, we entered into a letter of intent (the "Letter of Intent") with the University of Texas MD Anderson Cancer Center ("MD Anderson") pursuant to which MD Anderson shall receive a research grant from us entitled, "Validation of biomarker isomeso for pancreatic cancer," which is aimed at discovering new cancer-specific antigen targets (the "Grant"). The total costs to us to be paid in connection with the Grant shall be $300,000. Pursuant to the Letter of Intent, the Grant shall commence on April 1, 2021 and end on March 31, 2022.
Upcoming Investigational New Drug Application Submissions – We are planning to submit two investigational new drug ("IND") applications to the United States Food and Drug Administration by the end of May 2021. These INDs will be for our ALEXIS-PRO-1 and ALEXIS-ISO-1 product candidates. ALEXIS-PRO-1 is our allogeneic gamma delta chimeric T cell therapy product candidate targeting PD-L1. ALEXIS-ISO-1 is our allogenic gamma delta CAR-T cell therapy product candidate targeting Isomesothelin (the isoform of Mesothelin).
Q1 2021 Financial Highlights

Cash Position: Cash and cash equivalents were $7,335,300 as of March 31, 2021, compared to $10,150,500 as of December 31, 2020. The difference is attributable to cash outflows of $2,635,900, $44,700, and $134,600 for operating activities, investing activities, and financing activities respectively.

R&D Expenses: Our research and development expenses increased by $857,500, (83.41%), to $1,885,600 for the three months ended March 31, 2021. The increase was attributable to increased headcount, manufacturing, and experimentation costs for our ALEXIS-ISO-1 product candidate.

G&A Expenses: Our general and administrative expenses increased by $1,246,400, (151.15%), to $2,071,000 for the three months ended March 31, 2021 from $824,600 for the three months ended March 31, 2020. This increase was primarily due to increased headcount, stock compensation expenses from prior year grant modifications, and professional services.

Net Loss: Our net loss increased to $3,854,500 during the three months ended March 31, 2021 compared to $1,852,700 during the three months ended March 31, 2020.

On May 14, 2021 argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer, reported financial results for the first quarter 2021 and provided a business update (Press release, argenx, MAY 14, 2021, View Source [SID1234579972]).

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"We’ve had a strong start to 2021 with the acceptance for review of the BLA and J-MAA for efgartigimod in gMG by the regulatory agencies in the U.S. and Japan. The submissions in China and the EU are on track and we are well-positioned for a global launch of our first-in-class FcRn antagonist. We are building an exceptional team with significant launch experience in neurology and rare disease and hope to reach patients this year," said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

"Efgartigimod has the potential to help people living with gMG as well as several other severe autoimmune diseases mediated by IgG autoantibodies. Our team is advancing registrational trials across four indications with plans to start enrollment in two additional efgartigimod indications this year. We are also broadening our reach within autoimmunity with our first-in-class C2 inhibitor, ARGX-117, from which we will have Phase 1 data mid-year. To complement our clinical pipeline, we continue to invest in our discovery capabilities through our Immunology Innovation Program and strategic technology partnerships that position us well to generate long-term value for shareholders. We are closer each day to building an integrated, innovative, global immunology organization with the goal of impacting the lives of patients," concluded Mr. Van Hauwermeiren.

FIRST QUARTER 2021 AND RECENT BUSINESS UPDATE

Commercial preparations on-track for global launch of IV efgartigimod for gMG, including regulatory submissions, initial salesforce hires and key stakeholder engagement efforts

Biologics License Application (BLA) for IV efgartigimod for treatment of gMG accepted for review by U.S. Food and Drug Administration (FDA) with target action date of December 17, 2021 under Prescription Drug User Fee Act (PDUFA)
J-MAA submitted to Japan’s PMDA and accepted for review with anticipated Japan commercial launch in 2022
MAA expected to be filed with European Medicines Agency (EMA) in second half of 2021
Zai Lab Limited to discuss potential accelerated regulatory pathway for approval in China with National Medical Products Administration (NMPA)
Commercial readiness activities on-track, including:
Continued build-out of global commercial organization, including hiring of U.S. regional business directors during first quarter
Launched pre-approval access (PAA) program in March 2021 in U.S., Canada and Europe to open availability of efgartigimod to people living with gMG who meet the pre-approval access program criteria

Immunology pipeline advancing with five ongoing registrational trials of efgartigimod and initial upcoming data from second potential pipeline-in a product candidate, ARGX-117

Enrollment ongoing in five registrational trials across four indications, including ADAPT-SC (gMG), ADHERE (chronic inflammatory demyelinating polyneuropathy or CIDP), ADVANCE and ADVANCE-SC (primary immune thrombocytopenia or ITP), and ADDRESS (pemphigus)
Go-forward decision confirmed in February 2021 in ADHERE trial evaluating subcutaneous (SC) efgartigimod in CIDP based on evaluation of interim safety and efficacy assessments that surpassed pre-defined threshold
Enrollment in trials for fifth and sixth indications to begin in 2021
Additional efgartigimod indications to be evaluated as part of collaboration with Zai Lab Limited
Data expected mid-year from Phase 1 trial of C2 inhibitor, ARGX-117; Phase 2 dosing plan to be identified for indications, including multifocal motor neuropathy (MMN)
Phase 2 trial of MMN on track to start by end of 2021
Combination trials of cusatuzumab remain ongoing for treatment of acute myeloid leukemia (AML) as part of global collaboration and licensing agreement with Cilag GmbH International, an affiliate of Janssen
Decision to initiate additional cusatuzumab studies under collaboration will be determined following review of all available data
Immunology Innovation Program (IIP) continues to grow pipeline through wholly-owned development, partnered opportunities, asset-centric spinoff companies and the addition of strategic technology capabilities

Preclinical work ongoing in early-stage pipeline, including ARGX-118, ARGX-119 and ARGX-120
15-20 discovery programs under evaluation at any point in time that have emerged from IIP
Initiated collaboration and license agreement with Elektrofi to explore new subcutaneous formulations for current and future pipeline candidates, including efgartigimod
Secured exclusivity for FcRn and one additional target
Ongoing development of ARGX-112 (LEO Pharma), ARGX-114 (AgomAb), ARGX-115 (ABBV-151, AbbVie) and ARGX-116 (Staten Biotech) by IIP collaboration partners

Strong balance sheet and expanded Board of Directors support transition into integrated, global immunology organization

Completed public offering of 3,593,750 ordinary shares in February 2021 with gross proceeds of $1.15 billion
Implemented transition agreement for Chief Financial Officer Eric Castaldi as part of evolution to commercial-stage company; recruitment efforts ongoing for U.S.-based successor
Proposed resolutions presented during Annual General Meeting of Shareholders were approved, including:
Appointment to Board of Directors of Yvonne Greenstreet, President and Chief Operating Officer of Alnylam
Re-appointment of Anthony Rosenberg to Board of Directors
Approval of new remuneration policy
argenx to host virtual R&D Day on July 20, 2021 to share long-term corporate vision, disclose additional potential efgartigimod indications and provide updates across immunology pipeline.

As of January 1, 2021, the Company changed its functional and presentation currency from euro to U.S. dollars, which results in reporting its financial highlights in U.S. dollar as compared to euro in prior periods. Historical financials have been converted at the average exchange rate of the related period.

Cash, cash equivalents and current financial assets totaled $2,907.4 million on March 31, 2021, compared to $1,996.5 million on December 31, 2020. The increase in cash, cash equivalents and current financial assets resulted primarily from (i) the closing of a global offering, which resulted in the receipt of $1,092.1 million in net proceeds in February 2021, (ii) the net receipt of a $73.1 million non-creditable, non-refundable development cost-sharing payment in the form of newly issued Zai Lab shares received as part of the strategic collaboration for efgartigimod in Greater China, partially offset by (iii) the payment of $98.0 million related to the purchase of a priority review voucher from Bayer HealthCare Pharmaceuticals and other net cash flows used in operating activities.

Total operating income increased by $141.6 million for the three months ended March 31, 2021 to $167.4 million, compared to $25.8 million for the three months ended March 31, 2020. The increase was primarily due to the closing of the strategic collaboration for efgartigimod with Zai Lab, resulting in the recognition of $151.9 million in collaboration revenue.

Research and development expenses increased by $17.7 million for the three months ended March 31, 2021 to $122.3 million, compared to $104.7 million for the three months ended March 31, 2020. The increase in the first three months of 2021 resulted primarily from higher external research and development expenses, mainly related to the efgartigimod program in multiple indications and other clinical and preclinical programs. Furthermore, the research and development personnel expenses increased due to a planned increase in headcount and the increased costs of the share-based payment compensation plans related to the grant of stock options.

Selling, general and administrative expenses totaled $56.3 million for the three months ended March 31, 2021, compared to $27.6 million for the three months ended March 31, 2020. The increase resulted primarily from higher personnel expenses, including the costs of the share-based payment compensation plans related to the grant of stock options, and consulting fees linked to the preparation of a possible future commercialization of efgartigimod.

The increase in fair value on non-current financial assets amounted to $11.2 million for the three months ended March 31, 2021, which is the result of the closing of a Series B financing round of AgomAb Therapeutics, for which the Company maintains a profit share in exchange for granting the license for the use of HGF-mimetic antibodies from the SIMPLE Antibody platform.

Exchange losses totaled $28.8 million for the three months ended March 31, 2021, compared to an exchange gain of $23.0 million for the three months ended March 31, 2020. As a result of the change in the Company’s functional and presentation currency, the exchange losses for the three months ended March 31, 2021 are reflecting the unfavorable change in euro/U.S. dollar exchange rate, mainly attributable to unrealized exchange rate losses on cash, cash equivalents and current financial asset position in euro.

FINANCIAL GUIDANCE

Based on current plans to fund anticipated operating expenses and capital expenditures, argenx continues to expect its 2021 cash burn to approximately double from 2020. The increased spend will support the Company’s transition to an integrated immunology company, including the build-out of global commercial infrastructure and drug product inventory ahead of the expected launch of efgartigimod in gMG in the U.S, the advancement of its clinical-stage pipeline, including seven expected global trials of efgartigimod, and the continued investment in its Immunology Innovation Program.

EXPECTED 2021 FINANCIAL CALENDAR

July 29, 2021: HY 2021 financial results and business update
October 28, 2021: Q3 2021 financial results and business update
CONFERENCE CALL DETAILS
The first quarter 2021 financial results and business update will be discussed during a conference call and webcast presentation today at 2:30 pm CEST/8:30 am ET. A webcast of the live call may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website.

On May 14, 2021 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, reported its financial results for the first quarter ended March 31, 2021 and provided an update on recent corporate developments (Press release, Bio-Path Holdings, MAY 14, 2021, View Source [SID1234580002]).

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"The start of 2021 has been marked by substantial progress across our portfolio of targeted nucleic cancer drugs which included both presented and published data in support of our DNAbilize platform," stated Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings. "We recently announced the successful completion of the safety run-in of Stage 2 of the Phase 2 clinical study of prexigebersen for the treatment of acute myeloid leukemia (AML) in combination with frontline therapies, decitabine and venetoclax. This was particularly important as the efficacy portion of this study will include de novo fragile AML patients for whom a clean safety profile will be critical."

"Last month, we were particularly pleased to have supportive preclinical data from our BP1002 program presented before an audience of world-leading cancer specialists at the AACR (Free AACR Whitepaper) Annual Meeting and to have published an analysis highlighting the potential of prexigebersen within the oligonucleotide drug delivery landscape in the peer-reviewed journal, Biomedicines. This presentation and publication significantly enhanced the oncology community’s awareness of the potential for our DNAbilize platform in a variety of hard to treat cancers and we look forward to building on this momentum throughout the remainder of this year," continued Mr. Nielsen.

Recent Corporate Highlights

Announced Publication in Biomedicines. In April, Bio-Path announced the publication of an analysis highlighting the potential of prexigebersen (BP1001) within the antisense oligonucleotide drug delivery landscape in the peer-reviewed journal, Biomedicines.

Presented BP1002 Data at 2021 AACR (Free AACR Whitepaper) Annual Meeting. In April, Bio-Path presented a poster highlighting preclinical BP1002 data at the 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. BP1002 targets the protein Bcl-2, which is responsible for driving cell survival in up to 60% of all cancers. High expression of Bcl-2 has been correlated with poor prognosis for patients diagnosed with AML. The data presented in the AACR (Free AACR Whitepaper) poster show that venetoclax-resistant cells are sensitive to the inhibitory effects of BP1002 combined with decitabine, suggesting that this combination is a potential treatment for patients who have relapsed from frontline venetoclax-based therapies.

Successfully Completed Safety Cohort of Triple Combination in Stage 2 of Phase 2 Clinical Trial in AML. In April, Bio-Path announced the successful completion of the safety run-in of Stage 2 of the Phase 2 clinical study of prexigebersen (BP1001), a liposomal Grb2 antisense, for the treatment of acute myeloid leukemia (AML), in combination with frontline therapies, decitabine and venetoclax, in acute myeloid leukemia (AML) patients. The safety run-in of Stage 2 of the Phase 2 clinical trial was comprised of six evaluable patients who were treated with the triple combination of prexigebersen, decitabine and venetoclax.
Raised $13.0 Million in Public Offering. In February, Bio-Path announced the closing of a public offering for 1,710,600 shares of common stock at a price of $7.60 per share, for aggregate gross proceeds to the Company of approximately $13.0 million, before deducting the fees and estimated offering expenses payable by the Company.

Received Third U.S. Patent Grant Related to Manufacture of Platform Technology. In February, Bio-Path announced that the United States Patent and Trademark Office granted U.S. Patent No. 10,898,506 titled, "P-ethoxy nucleic acids for liposomal formulation." The new patent builds on earlier patents granted that protect the platform technology for DNAbilize, the Company’s novel RNAi nanoparticle drug.
Financial Results for the First Quarter Ended March 31, 2021

The Company reported a net loss of $2.4 million, or $0.43 per share, for the three months ended March 31, 2021, compared to a net loss of $3.3 million, or $0.90 per share, for the three months ended March 31, 2020.

Research and development expense for the three months ended March 31, 2021 decreased to $1.3 million, compared to $2.0 million for the three months ended March 31, 2020 primarily due to decreased preclinical expense related to timing of activities for BP1003 as well as decreased clinical trial expense due to timing of activities for our Phase 2 clinical trial of prexigebersen in AML and our Phase 1 clinical trial of BP1002 in lymphoma.

General and administrative expense for the three months ended March 31, 2021 decreased to $1.2 million, compared to $1.3 million for the three months ended March 31, 2020 primarily due to decreased franchise tax expense.

As of March 31, 2021, the Company had cash of $30.8 million, compared to $13.8 million as of December 31, 2020. Net cash used in operating activities for the three months ended March 31, 2021 was $1.6 million compared to $2.5 million for the comparable period in 2020. Net cash provided by financing activities for the three months ended March 31, 2021 was $18.6 million.
Conference Call and Webcast Information

Bio-Path Holdings will host a conference call and webcast today at 8:30 a.m. ET to review these first quarter 2021 financial results and to provide a general update on the Company. To access the conference call please dial (844) 815-4963 (domestic) or (210) 229-8838 (international) and refer to the conference ID 5064037. A live audio webcast of the call and the archived webcast will be available in the Media section of the Company’s website at www.biopathholdings.com.

On May 14, 2021 Biotech company Prescient Therapeutics (ASX:PTX) reported that strengthening its ties in research and development with the world-renowned Peter MacCallum Cancer Centre (Peter Mac) (Press release, Prescient Therapeutics, MAY 14, 2021, View Source;utm_medium=rss&utm_campaign=prescient-strengthens-ties-with-leading-car-t-research-centre [SID1234580018]).

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Based in Melbourne, the Peter Mac centre is at the cutting edge of research and manufacturing advancements in CAR-T therapies – considered a breakthrough treatment in the fight against cancer.

The deal will see PTX get direct access to world-class facilities and a team of CAR-T experts led by Professor Phil Darcy, who will assist in the pre-clinical development of Prescient’s OmniCAR programs.

In that context, extending its partnership with the Peter Mac centre provides the ideal environment for further development of its next-generation OmniCAR programs.

Development opportunity
Prescient is currently developing three OmniCAR platforms used in the treatment of acute myeloid leukaemia (AML), Her2+ solid tumours and glioblastoma multiforme (GBM) – an aggressive form of cancer that usually begins in the brain.

The company is developing OminCAR "as a next generation CAR-T platform, capable of being deployed by other CAR-T and oncology companies under licence to advance their own programmes".

Known as Chimeric Antigen Receptor T-cells, CAR-T cells are specifically engineered to hunt and destroy abnormal cells, including cancerous cells.

And as part of its agreement with Peter Mac, it will now have two post-doctoral scientists and two research assistants working full-time on the development of its research suite.

"Prescient has also secured grant funding of $100,000 from the Federal Government’s Innovation Connections scheme towards this research," the company highlighted.

For Darcy, the use of Prescient’s OmniCAR treatment presents a pathway to achieve the replication of breakthrough CAR-T treatments in solid tumours.

"We are excited by the opportunity and potential offered by the OmniCAR platform to make headway into solid tumours and other blood cancers, and to greatly enhance and improve the clinical control and efficacy of existing CAR-T cancer therapies," Darcy said.

While PTX will get access to the Peter Mac centre’s leading team of researchers and scientists, it will fully own any intellectual property that arises from work carried out.

Prescient managing director Steven Yatomi-Clarke said the deal is reflective of the company’s strong medical networks, as it builds out its OmniCAR research programs "to the highest standard".

"This latest research program is an important part of Prescient’s development plans, which include institutional and commercial laboratories," he said.

"We continue to work very closely with Professor Darcy and the team at Peter Mac, as we progress this exciting development of controllable and adaptable next generation CAR-T therapies."

On May 14, 2021 Gilead Sciences, Inc. (Nasdaq: GILD) reported that its executives will be speaking at the following investor conferences (Press release, Gilead Sciences, MAY 14, 2021, View Source [SID1234580050]):

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Cowen Annual Virtual Oncology Innovation Summit on Friday, May 21 at 12:00pm ET
Jefferies Virtual Healthcare Conference on Tuesday, June 1 at 1:30pm ET
Bernstein Annual Strategic Decisions Conference on Wednesday, June 2 at 4:30pm ET
Goldman Sachs Annual Global Healthcare Conference on Wednesday, June 9 at 2:10pm ET
The live webcasts can be accessed at the company’s investors page at investors.gilead.com. The replays will be available for at least 30 days following the presentation.